ClinicalTrials.gg
RecruitingNCT06309979Updated Jun 23, 2026

A Study to Assess Growth in Children With Idiopathic Short Stature

An observational study in Idiopathic Short Stature, sponsored by BioMarin Pharmaceutical. Recruiting at 38 sites in United States. Open to participants aged 2 Years to 16 Years. Per ClinicalTrials.gov, last updated 2026-06-23.

Sponsored by BioMarin Pharmaceutical · Observational

From the registry’s dates

  • Started Aug 2024; still recruiting 2 years 3 months later.
Study type
Observational
Model
Cohort
Time perspective
Other
Enrollment
300
Ages
2 Years to 16 Years
Sex
All
01

Study summary

Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.

02

Conditions studied

  • Idiopathic Short Stature
03

In context

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 16 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

The study population will include up to 600 pediatric individuals with Idiopathic Short Stature.

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Participants must be > 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the .
  2. A height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth chart (https://www.cdc.gov/growthcharts/zscore.htm).
  3. Participants who have either never received hGH, or who are currently receiving hGH treatment.
  4. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
  5. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.

Exclusion criteria

Exclusion Criteria:

  1. Diagnosis of systemic disease or condition that may cause short stature, eg renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. Children with such diagnoses can be considered for inclusion if their condition is well controlled, at the discretion of the Medical Monitor.
  2. Known presence of one or more pituitary hormone deficiencies
  3. Bone age advanced over chronological age by more than 3 years.
  4. For hGH naïve participants, historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (between -1.00 SDs and +2.00 SDs).
  5. For participants currently on hGH treatment, historic results before GH treatment of stimulation test with serum or plasma GH level greater than 10 μg/L or serum IGF-1 test between -1.00 SDs and +2.00 SDs.
  6. Have received an product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Observational model
Cohort
Time perspective
Other
Enrollment
300 participants (estimated)
Patient registry
No
06

What researchers measure

Primary outcomes

  1. Change in annualized growth velocity (AGV)

    Time frame: Every 6 months through end of study, up to 15 years

  2. Change in Height Z-score

    Time frame: Every 6 months through end of study, up to 15 years

  3. Change in Standing Height

    Time frame: Every 6 months through end of study, up to 15 years

  4. Change in BMI

    Time frame: Every 6 months through end of study, up to 15 years

  5. Change in BMI Z-score

    Time frame: Every 6 months through end of study, up to 15 years

Secondary outcomes

  1. Frequency of event rates of medical events of interest

    Time frame: Every 6 months through end of study, up to 15 years

  2. Frequency of Serious Medical Events

    Time frame: Every 6 months through end of study, up to 15 years

  3. Association between specific variants and growth velocity

    Time frame: Every 6 months through end of study, up to 15 years

  4. Association between other health outcomes directly related to short stature

    Time frame: Every 6 months through end of study, up to 15 years

07

Study locations

21 of 38 sites recruiting
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 23, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06309979
Responsible party
Sponsor
First posted
Mar 13, 2024
Start date
Aug 8, 2024
Primary completion
Dec 31, 2040 (estimated)
Completion
Dec 31, 2040 (estimated)
Last update
Jun 23, 2026

Study contacts

Trial Specialist
Contact
Medinfo@bmrn.com
18009834587

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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